The star of the acquisition is the enzyme replacement therapy INZ-701, being developed for the rare disease ENPP1 deficiency.
BioMarin is acquiring rare disease specialist Inozyme Pharma for $270 million to build out its portfolio of enzyme therapies, the biotech announced on Friday.
BioMarin will pay $4 for each outstanding share of Inozyme. The companies expect to complete the transaction in the third quarter, pending customary closing conditions such as anti-trust clearances and the tender of at least a majority of Inozyme shares. The Board of Inozyme has unanimously recommended that stockholders tender their shares.
The centerpiece of Friday’s deal is Inozyme’s INZ-701, an investigational enzyme replacement therapy currently in late-stage development for ectonucleotide pyrophosphatase/phosphodiesterase 1 (ENPP1) deficiency. Caused by mutations to the ENPP1 gene, ENPP1 deficiency is a rare and progressive disease that can affect a variety of organs, presenting differently from patient to patient.
Common symptoms include early-onset osteoporosis, a heightened risk of fractures and calcification of the tendons. Patients with ENPP1 deficiency are also more susceptible to cardiovascular mortality, severe rickets and softening of the bones. There are currently no approved treatments for the condition.
Delivered subcutaneously, INZ-701 combines the active site of the ENPP1 enzyme with a specific fragment of a human antibody, according to Inozyme’s website. This structure allows INZ-701 to be distributed throughout the body, essentially replacing the deficient ENPP1 enzyme and carrying out its function. Preclinical data have supported this mechanism of action, showing that INZ-071 could prevent abnormal mineralization.
Aside from ENPP1 deficiency, Inozyme is also testing INZ-701 for ABCC6 deficiency and calcific uremic arteriopathy, both rare diseases.
The acquisition comes after some rocky times for BioMarin. Last year the company reconfigured its c-suite, laid off 225 employees and axed four molecules as part of a broader reorganization plan.
“As BioMarin continues our transformation and delivers on our corporate strategy, we will continue to evaluate external innovation alongside internal innovation,” said BioMarin’s president and CEO Alexander Hardy in a statement announcing the deal. “We are in a strong financial position to bring in additional assets as we accelerate the development of medicines for patients with significant unmet need.”
The Inozyme acquisition also follows BioMarin’s first-quarter earnings call, where Hardy said that the biotech would conduct “at least one business development deal this year.” BioMarin made $745 million in Q1, which represents a 15% year-on-year increase and was ahead of the analyst consensus of $738.1 million.
BioMarin’s enzyme therapy portfolio accounted for much of its topline, bringing in $484 million in the quarter, an 8% jump from the same period last year. Voxzogo, its closely watched C-type natriuretic peptide analog indicated for achondroplasia with open epiphyses, surged 40% year-on-year to bring in $214 million.
With a strong start to 2025, BioMarin reaffirmed its full-year outlook. The company expects total revenues to hit $3.1 billion, with Voxzogo contributing at least $900 million.
Still, analysts at William Blair remain cautious about BioMarin. In a May 2 note to investors, the analysts flagged “uncertainty” regarding the biotech’s “current revenue drivers” and whether they have “the potential to continue delivering significant value creation in the near term.”
